News

A Phase 3 study of Sollpura (liprotamase), a non-porcine enzyme replacement therapy aiming to treat exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF) better than existing porcine-derived therapies, is now recruiting about 150 pediatric and adult patients in the United States, Europe and Israel. Specifically, the expanded trial will evaluate the efficacy — or non-inferiority — of Sollpura compared…

A deeper understanding of how bacteria like Pseudomonas aeruginosa acts and survives in people despite antibiotic treatment was the focus of a doctoral thesis, along with a recommendation for antibiotic combinations that might work better those what’s currently in use. The thesis, “Host-pathogen interactions in Pseudomonas aeruginosa invasive and respiratory tract infection,” is by…

A computer program created by physicians in Cincinnati may help cystic fibrosis (CF) patients to keep using those at-home treatments they feel are most effective, by providing them an easy way of sharing their preferences — and a given therapy’s effectiveness — with their doctors. CF patients rely on a variety of home therapies, from…

Proteostasis Therapeutics has filed an Investigational New Drug application with the U.S. Food and Drug Administration for PTI-808 as a treatment for cystic fibrosis. PTI-808 promotes the activity of the cystic fibrosis transmembrane conductance regulator, or CFTR, the protein that is faulty in CF. Scientists call the drug a potentiator…

Cystic Fibrosis Foundation Therapeutics (CFFT) will increase funding to support Spyryx Biosciences‘ Phase 2 trial assessing SPX-101 in people with cystic fibrosis. The funding is part of an award Spyryx received in 2015 from CFFT, the Cystic Fibrosis Foundation‘s…

Children with cystic fibrosis (CF) have less sleep and more sleep interruptions than healthy children, even when the disease is well-managed and stable, an Australian study indicates. The research, “How Well Do Children with Cystic Fibrosis Sleep? An Actigraphic and Questionnaire-Based Study,” was published in The Journal of…

Forty cystic fibrosis patients or family members are winners of Vertex Pharmaceuticals‘ first All in for CF scholarships. The awards are for $5,000, making the total for the 2017-2018 academic year $200,000. Boston-based Vertex is a biotech company that develops therapies for cystic fibrosis and  other life-threatening conditions. The company…

So-called silent gene mutations in the cystic fibrosis (CF)-causing CFTR gene are not necessarily silent at all. A research team discovered that one such mutation also causes the resulting protein to have a poor function. The insights put the spotlight on the complexity of CF genetic causes, but also advances…

Abiding by the extensive treatment regimen for cystic fibrosis (CF) can be especially challenging for pediatric patients. A new study found that frequent monitoring of pulmonary function tests (PFTs) and medication reminders can help pediatric CF patients better meet their daily medication and vitamin…

Celtaxsys announced it has completed enrollment of its Phase 2b clinical trial evaluating the company’s lead anti-inflammatory candidate, oral acebilustat, in adult patients with cystic fibrosis (CF). Acebilustat (formerly known as CTX-4430) is a once-daily, oral anti-inflammatory being evaluated for its safety and efficacy in treating diseases like CF. It is a new small…